Sarepta's confirmatory Duchenne trial fails to meet endpoint, complicating future for controversial drug
Sarepta and Roche’s confirmatory study for a Duchenne gene therapy failed to hit its primary endpoint, complicating plans to convert the drug’s label into a full approval for a wider population of boys.
In the Phase III EMBARK study, 125 patients received either placebo or Elevidys, the gene therapy developed by Sarepta. After one year, those who received Elevidys saw a 2.6-point improvement compared to a 1.9-point improvement in the placebo group on a functional mobility scale called the North Star Ambulatory Assessment, the companies said Monday.
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